Wave Life Sciences Announces Positive Phase 1 Results for WVE-210201 in Duchenne Muscular Dystrophy (DMD)

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CAMBRIDGE, Mass., Dec. 06, 2018 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (NASDAQ: WVE), a biotechnology company focused on delivering transformational therapies for patients with serious, genetically-defined diseases, today announced that the safety and tolerability data from the WVE-210201 Phase 1 clinical trial in boys with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping support initiation of a Phase 2/3 clinical trial. Based on results from four ascending dose cohorts in the Phase 1 trial and pending final analysis, Wave has selected a dose for its planned Phase 2/3 clinical trial of WVE-210201, which it intends to initiate in 2019. In parallel, the independent Safety Monitoring Committee of the Phase 1 clinical trial has endorsed moving forward with a higher dose of WVE-210201 to be studied in a fifth cohort. Wave plans to present the results from the WVE-210201 Phase 1 clinical trial, as well as details of the Phase 2/3 study design, at upcoming scientific meetings.

“These results mark an important milestone for the Duchenne community and toward our goal of inducing meaningful, natural dystrophin expression in boys with DMD who are amenable to exon 51 skipping,” said Paul Bolno, MD, MBA, President and Chief Executive Officer of Wave Life Sciences. “This is an exciting time at Wave as we continue to partner with the patient and medical communities to advance our lead program in DMD. We thank all of the boys and their families who are participating in this Phase 1 trial and its open-label extension and are grateful for their trust and commitment.”

WVE-210201 is also currently being studied in an ongoing multi-dose, open-label extension (OLE) study initiated in August 2018 and available to patients as they complete the Phase 1 clinical trial. The company remains on track to deliver an interim analysis of dystrophin expression from muscle biopsies in boys receiving WVE-210201 in the OLE study in the second half of 2019. Data from this analysis are intended to be an important component of a submission to the U.S. Food and Drug Administration for accelerated approval.

“PPMD continues to be optimistic about the progress the team at Wave Life Sciences is making with their stereopure exon 51 skipping program for Duchenne. We are pleased to see WVE-210201 advance on the clinical development path and look forward to more updates from the Wave team,” said Pat Furlong, founding President and CEO of Parent Project Muscular Dystrophy (PPMD).

Wave anticipates initiating a global, placebo-controlled Phase 2/3 efficacy and safety clinical trial of WVE-210201 in DMD patients amenable to exon 51 skipping in 2019. The Phase 2/3 trial is designed to measure clinical efficacy and dystrophin expression, and the company intends to use the results of this trial to seek regulatory approvals globally.

Graham Morrell781-686-9600

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Globe Newswire: 12:30 GMT Thursday 6th December 2018

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